Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Read our disclaimer for details.

Pharmacokinetics including half-life and concentration of GMI-1070 in the blood and urine

Markers of inflammation and cell stickiness in the blood [ Time Frame: Up thru 28 days post last dose ]

Original Secondary Outcome Measures ICMJE

Same as current

Current Other Outcome Measures ICMJE

Not Provided

Original Other Outcome Measures ICMJE

Not Provided

Descriptive Information

Brief Title ICMJE

Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

Official Title ICMJE

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Pharmacokinetics of GMI-1070, A Pan-Selectin Inhibitor, In Subjects Hospitalized For Sickle Cell Vaso-Occlusive Crisis

Brief Summary

GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also known as a sickle cell pain crisis). The study will also collect information on the safety of GMI-1070, how much of the drug is in the blood and urine, and if there are any other effects when used in patients who are in the hospital for a sickle cell pain crisis.

Detailed Description

Patients being admitted to the hospital for pain crisis may be eligible for this study. In addition, patients should be 12-60 years old and have sickle cell types SS or S-beta-thalassemia. People who take part in the study will be evaluated and then randomly assigned to receive either GMI-1070 or a placebo by IV, in addition to all other usual treatments for their pain crisis.

During the hospital stay for pain crisis, GMI-1070 or placebo will be given twice a day, and patients will be asked about their pain severity (pain score) at the beginning of the study and every few hours during their hospital stay. Their general health, vital signs, lab tests, and pain medications will also be checked on a regular basis through the hospital stay. When a patient is feeling well enough to go home, the study drug (GMI-1070 or placebo) will be stopped, and the patient may go home. Participants will be asked to come back to clinic for a check-up a few days after leaving the hospital, and one month after leaving the hospital.

Hospitalization for uncomplicated VOC, or treated with parenteral pain medications in other medical settings such as the emergency department or day hospital for uncomplicated VOC, within past 14 days.

o Subjects may be included if treated as an outpatient within the past 48 hours for the same VOC episode.

Systemic steroid therapy within 48 hours prior to enrollment or expectation that therapy may be used during the study (inhaled or topical steroids are allowed)

For those on chronic or long-acting opioids, a change in dose in the past 14 days OR pain requiring medical attention in the past 14 days (change in opioid medication for acute pain in the past 48 hours and directly related to this VOC admission is allowed)

Greater than 5 episodes of hospitalization for VOC in the past 6 months (180 days)

Medical or psychiatric condition that, in the opinion of the investigator, may pose a risk to the subject for participation or interfere with the conduct or results of the study

Currently receiving, or has received within the previous 4 weeks, any other investigational agent

Previous administration of GMI-1070

Expectation that the subject will not be able to be followed for the duration of the study

Pregnant or lactating female; or female of childbearing potential or male unable or unwilling to comply with birth control methods or abstinence during the course of the study

Active use of illicit drugs and/or alcohol dependence, as determined by the investigator

Sex/Gender

Sexes Eligible for Study:

All

Ages

12 Years to 60 Years (Child, Adult)

Accepts Healthy Volunteers

No

Contacts ICMJE

Contact information is only displayed when the study is recruiting subjects